Non-toxic Neuro Disease Therapy

Track Code: 
16KU025M
Summary: 

The current invention concerns the novel use of an endogenous estrogen derivative that has a super-strong neuroprotective effect against oxidative neurotoxicity in vivo.

Overview: 

It is well established that oxidative stress is one of the etiological factors in the development of neurodegenerative diseases including Alzheimer's. A number of studies show that estrogens can be produced in the brain from steroid precursors, and estrogens have been found to have neuroprotective functions.

In the proposed invention, the inventors discovered that a unique endogenous estrogen derivative that has a super-strong protective effect against neuronal cell death both in cultured neuronal cells and in animal models in vivo. In addition, they have developed a novel explanation for the molecular mechanism of the neuroprotective actions.

Applications: 

The inventors propose that the body uses certain endogenously-formed estrogen derivatives as special protectors against neuronal cell death, and consequently, these estrogen derivatives can be used as potential therapy in neurodegenerative disorders.

How it works: 

This endogenous estrogen derivative, which has little estrogenic activity, can be selectively formed in neuronal cells in the brain. This estrogen derivative, when used as a drug, has a super-strong neuroprotective effect against oxidative neurotoxicity both in vitro (neuronal cell culture) and in vivo (animal models). Its protective effect is much stronger than the classical estrogens 17beta-estradiol and estrone. Mechanistically, the neuroprotection by this endogenous estrogen derivative involves increased cytoplasmic translocation of p53 resulting from SIRT1-mediated deacetylation of p53.

Benefits: 

This endogenous estrogen derivative has little binding affinity for the estrogen receptors, and thus it has basically no estrogen's hormonal activity. As such, it will have minimal adverse effects compared to other estrogens such as 17beta-estradiol and estrone. We believe that this endogenous estrogen derivative may be successfully used as an effective treatment in neurodegenerative disorders.

Why it is better: 

Based on the doses needed in rats to exert this neuroprotective effect, it is possible that this endogenous estrogen derivative is one of the most potent neuroprotective agents known to date. In addition, this estrogen derivative has little estrogenic activity, and is expected to have minimal side effects in clinical settings.

Other Applications: 

Ischemic stroke; cancer biomarkers; Parkinson's disease; dementias; Alzheimer's disease.

Licensing Associate: 
Aswini Betha, PhD · abetha@ku.edu · 913-588-5713
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Keyword(s): 
Inventor(s): 
Bao-Ting Zhu